Palovarotene
- Generic Name
- Palovarotene
- Brand Names
- -
- Drug Type
- Small Molecule
- Chemical Formula
- C27H30N2O2
- CAS Number
- 410528-02-8
- Unique Ingredient Identifier
- 28K6I5M16G
- Background
Fibrodysplasia Ossificans Progressiva (FOP), with an estimated worldwide prevalence of one in 2 million individuals, is an exceptionally rare genetic disorder. FOP is caused by a gain-of-function mutation in the ACVR1/ALK2 gene which results in progressive heterotopic ossification, a process wherein connective tissues (e.g. skeletal muscle, ligaments, tendon...
- Indication
Palovarotene is indicated in Canada to reduce the formation of heterotopic ossification in adults and children (≥8 years old for females and ≥10 years old for males) with Fibrodysplasia Ossificans Progressiva (FOP) by Health Canada and FDA.
- Associated Conditions
- Heterotopic Ossification (HO)
- Associated Therapies
- -
A Rollover Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged ≥14 Years With Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed the Relevant Parent Studies.
- Conditions
- Interventions
- First Posted Date
- 2021-08-30
- Last Posted Date
- 2024-11-26
- Lead Sponsor
- Ipsen
- Target Recruit Count
- 63
- Registration Number
- NCT05027802
- Locations
- 🇦🇷
Hospital Italiano de Buenos Aires, Buenos Aires, Argentina
🇺🇸Mayo Clinic, Rochester, Minnesota, United States
🇺🇸Children's Hospital of Philidelphia, Philadelphia, Pennsylvania, United States
Study Evaluating the Effect of Food on the Pharmacokinetics of Palovarotene and the Effect of Palovarotene on the Pharmacokinetics of the CYP3A4 Substrate Midazolam in Two Cohorts of Healthy Adult Subjects
- Conditions
- Interventions
- First Posted Date
- 2021-04-02
- Last Posted Date
- 2021-04-02
- Lead Sponsor
- Clementia Pharmaceuticals Inc.
- Target Recruit Count
- 48
- Registration Number
- NCT04829773
- Locations
- 🇺🇸
Cambridge Ipsen US, Cambridge, Massachusetts, United States
Study to Assess Safety, Tolerability, and Pharmacokinetics of Single and Multiple Ascending Doses of Palovarotene Ophthalmic Solution in Healthy Adult Subjects
- First Posted Date
- 2021-02-21
- Last Posted Date
- 2021-02-21
- Lead Sponsor
- Clementia Pharmaceuticals Inc.
- Target Recruit Count
- 48
- Registration Number
- NCT04762355
- Locations
- 🇨🇦
Algorithme Pharma facility, Québec, Canada
An Efficacy and Safety Study of Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva.
- First Posted Date
- 2017-10-18
- Last Posted Date
- 2023-11-29
- Lead Sponsor
- Clementia Pharmaceuticals Inc.
- Target Recruit Count
- 107
- Registration Number
- NCT03312634
- Locations
- 🇦🇷
Hospital Italiano de Buenos Aires, Tte General Juan Domingo Peron 4190, Buenos Aires, Argentina
🇬🇧Royal National Orthopaedic Hospital, Brockely Hill, Stanmore, United Kingdom
🇦🇺Royal North Shore Hospital, Saint Leonards, New South Wales, Australia
In-Home Evaluation of Episodic Administration of Palovarotene in Fibrodysplasia Ossificans Progressiva (FOP) Subjects
- First Posted Date
- 2015-08-13
- Last Posted Date
- 2020-10-19
- Lead Sponsor
- Clementia Pharmaceuticals Inc.
- Target Recruit Count
- 6
- Registration Number
- NCT02521792
- Locations
- 🇺🇸
University of Pennsylvania, Center for Research in FOP & Related Disorders, Philadelphia, Pennsylvania, United States
🇺🇸University of California San Francisco, Division of Endocrinology and Metabolism, San Francisco, California, United States
An Efficacy and Safety Study of Palovarotene to Treat Preosseous Flare-ups in FOP Subjects
- Conditions
- Interventions
- First Posted Date
- 2014-07-15
- Last Posted Date
- 2021-02-16
- Lead Sponsor
- Clementia Pharmaceuticals Inc.
- Target Recruit Count
- 40
- Registration Number
- NCT02190747
- Locations
- 🇺🇸
University of California San Francisco, Division of Endocrinology and Metabolism, San Francisco, California, United States
🇫🇷Hôpital Necker-Enfants Malades, Department of Genetics, Paris, France
🇬🇧The Royal National Orthopaedic Hospital, Brockley Hill, Stanmore, Middlesex, United Kingdom
Drug Development Updates
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